TPAC的动态

I'm sure many of you have seen the recent celebrated announcement of FDA's approval of Lenmeldy, a gene therapy that completely changes the paradigm for #MLD, a truly devastating and life-limiting disease. You are also probably starting to see the price tag, $4.25 million. And maybe wincing. But it's important for us to dive into how these price tags come to be and the two articles linked below are fairly well written on this point and highlight the use of an ICER assessment in pricing. Though they, unfortunately, both succumb to the all too-easy framing of Lenmeldy as the “most expensive drug in the world.” But that’s a bit misleading, because Lenmeldy is potentially a one-time treatment, and there are numerous other chronic-disease drugs out there with lifetime costs that are far higher. Certainly, this will not be the last time we see another "most expensive" drug and it is most likely going to be another gene therapy with the potential to save lives and change the face of a disease. The question that we must all focus on now is how will we start to turn availability of therapeutic innovation into accessibility of therapeutic innovation? And the answer to this will most certainly require its own transformative solutions. https://lnkd.in/gB5cc4Qq https://lnkd.in/gjMWUZ23

How available are cell and gene therapies across the world? This chart shows the availability of accredited CAR T-cell treatment centers varies significantly by country and is concentrated in high-income countries, However, the chart also shows that there is more to the story. Many sites only have experience in a research setting, while much fewer have experience with commercially available CAR T therapies. ?? In the U.S., 818 clinical trial sites have worked with patients in trials testing CAR T-cell therapies, yet only 198 treatment centers are certified to treat patients with commercially available CAR Ts. ?? This shows the potential opportunity for treatment center expansion, but the resources needed to make that happen may be a barrier. ?? China has the second-largest number of trial experienced sites but few accredited treatment centers. ?? This is likely because there are currently only two commercially available CAR T-cell therapies in China. Download IQVIA Institute’s Strengthening Pathways for Cell and Gene Therapies report to read the rest of the story of cell and gene therapy: https://bit.ly/4bxOcxI. #CellTherapy #GeneTherapy

This is a super-interesting development to the gene therapy market access challenge. Years ago I worked on a project looking at how we might come up with a different system that benefits everyone involved (pharma, healthcare systems and patients) and came up with some exciting pilot ideas; would have been amazing to have seen a shift. (And if you are involved or interested in something like this, please do give me a shout!) Thoughts? Can we make gene therapy possible despite the cost? https://lnkd.in/eZDgX35M

Ophthopedia Update:Atsena completes dosing in part A of X-linked retinoschisis gene therapy trial: Atsena Therapeutics completed patient dosing in part A of the phase 1/2 LIGHTHOUSE clinical trial investigating a gene therapy designed for the treatment of X-linked retinoschisis, according to a press release. Part A of the trial is evaluating three dose levels of ATSN-201, a subretinal injection, in nine adults with X-linked retinoschisis (XLRS). The therapy uses AAV.SPR, the company’s novel spreading capsid, to reach therapeutic levels of gene expression in photoreceptors in the central retina, according to the release. The open-label, dose-escalation, dose-expansion LIGHTHOUSE trial #ophthalmology #eye #eyenews

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> FDA widens Sarepta’s Duchenne gene therapy label to older boys in significant expansion of use: The FDA has broadened the label for Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy to patients ages 4 and older. The approval goes beyond the patient population in which the company reported data for its randomized clinical trials. It includes a full approval for Duchenne patients who are not dependent on a wheelchair, and an accelerated approval for those who are wheelchair-dependent. Marketed as Elevidys, the gene therapy was initially given accelerated approval last year for boys ages 4 and 5 who have Duchenne muscular dystrophy. It’s meant to slow progression of the aggressive muscle disease, and the expansion to older boys has long been awaited by families of patients who have few treatment options. It costs $3.2 million in the US and is not approved anywhere else in the world. There have been signs the FDA was going to expand the therapy’s label. Earlier this year, CBER Director Peter Marks made comments suggesting he was open to doing so. And when Elevidys was initially approved, Marks overruled FDA reviewers who had recommended rejecting it. Marks again overruled FDA staff in broadening the label of Sarepta’s gene therapy. Memos that were released Thursday with the agency’s decision showed that two top FDA officials, Lola Fashoyin-Aje and Nicole Verdun, recommended rejecting the gene therapy in favor of an additional confirmatory study but were overruled by CBER chief Peter Marks. It’s been debated whether the evidence has been strong enough to back the expansion. In October, the company reported that the Phase 3 EMBARK study failed to meet its primary endpoint. Intended as a confirmatory study, it showed that boys ages 4 to 7 who got Elevidys didn’t do better on a 17-item motor skill test than those who got placebo. However, the study met key secondary endpoints — measuring boys’ ability to stand and walk — that Sarepta cited when asking the FDA to approve the therapy for boys with Duchenne regardless of their wheelchair status or age. Continued approval for non-ambulatory Duchenne patients may depend on results from a confirmatory trial, which Sarepta said would be the Phase 3 ENVISION study with non-ambulatory and older ambulatory people with Duchenne. In its first six months on the market, Elevidys generated $200 million in revenue, reflecting strong demand for the therapy. The company’s stock $SRPT was up 35% in after-hours trading. It’s also been helped by the failure of a potential competitor: Last week, Pfizer announced that its Duchenne gene therapy did not succeed in a Phase 3 study. Pfizer’s therapy did not meet any of the secondary endpoints that Sarepta pointed to when asking for its label expansion. #lucidquest #genetherapy #celltherapy

Have you inadvertently arrived in a treatment comfort zone? Cell and gene therapies are relatively new, and a lot of work is needed to promote their uptake. With patients, caregivers and physicians becoming entrenched in patterns of decision marking, there’s a real danger of people with rare and debilitating diseases missing out on treatments that can improve their quality of life. To learn how to design an effective medical communications campaign and how and when to nudge a target, read this recent article in Cell & Gene from William H (Alpharmaxim) and Lisa Campbell (SSI Strategy): https://lnkd.in/dhTXjsf9

?? ?????????? ???????? ?????????????? ???????????? Pfizer announces that its gene therapy has failed to improve motor function in boys aged 4-7 with Duchenne Muscular Dystrophy in a phase 3 trial. The therapy, ?????????????????????????????? ?????????????????????????(PF-06939926), missed secondary measures + trials were suspended after unfortunate patient deaths. ?? Pfizer is evaluating next steps for the program. Sarepta's Elevidys (delandistrogene moxeparvovec), the only FDA-approved gene therapy for DMD, also recently failed to meet its primary objective in a phase 3 trial. Despite this, they proceeded with an additional filing based on secondary endpoint efficacy.??????? ?????? ???? ???????????????? ???? ?????????????? ?? ?????????????? ???? ???????????????? ?????????? ???????? ??????????. ?? The findings from Pfizer's Phase 3 CIFFREO study on Duchenne Muscular Dystrophy highlight the challenges in developing treatments for this disease. Although the primary endpoint wasn't met, the insights gained will inform future research, moving us closer to effective therapies for DMD. #genetherapy #cellandgenetherapy #biotech #CGTweekly

Transformational gene therapy for haemophilia B available on the NHS! NHS England has announced that Hemgenix, a new gene therapy for moderately severe or severe haemophilia B, will be available on the NHS. This one-time therapy, which reduces the need for frequent hospital visits for medication, marks a significant advancement in treatment options. The transformational gene therapy for haemophilia B, with an official cost of ￡2.6m is now available in the UK! This revolutionary treatment offers hope and an improved quality of life for those living with the bleeding disorder. A patient who has received the new drug says he "feels cured" and the gene therapy has given him a "new lease of life". Professor Sir Stephen Powis, NHS National Medical Director, said: " It's the first drug to be made available in our Innovative Medicines Fund to provide early access for patients while further data is collected on its long-term benefits." "Amazing news for patients! Having worked to successfully launch a gene therapy in the UK, I know just how transformative the benefits can be to patients and their families. A reminder of why I go to work every day." Will Wright - Associate Director, HEOR and Access The recent authorisation of Hemgenix reminds us of the profound impact of our work at FIECON. We are dedicated to driving positive change in healthcare by ensuring that cost does not hinder access to life-saving treatments. As a leading Health Economics consultancy, we put patients at the heart of everything we do. We advance the healthcare industry by evaluating and supporting innovative therapies, ensuring they reach patients swiftly and effectively. #HealthEconomics #PatientFirst #haemophilia

Pfizer has announced that the results of its CIFFREO trial of mini-dystrophin gene therapy fordadistrogene movaparvovec to treat Duchenne muscular dystrophy (DMD) show that it didn’t meet its primary or secondary endpoints. CIFFREO was a randomized, double-blind, placebo-controlled study Phase 3 trial in ambulatory (walking) boys aged four to seven years of age with DMD. The primary endpoint in the study was improved motor function and assessed by a change in the North Star Ambulatory Assessment (NSAA) at one year after treatment. The key secondary endpoints included measuring the ten-meter run/walk velocity and time to rise from floor velocity. While gene therapy has brought a lot of hope for DMD, these results are very disappointing. There is still a lot more that we need to understand about gene therapy as a treatment for DMD and how it can be optimised as a treatment. We think this is a good time to bring the community together to discuss what has been learnt so far about gene therapy for DMD and what research is taking place to develop it. We are going to organize a webinar for the community, and you can email questions you’d like raised in it to [email protected].

Gene&Cell Therapy >> FDA widens Sarepta’s Duchenne gene therapy label to older boys in significant expansion of use: The FDA has broadened the label for Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy to patients ages 4 and older. The approval goes beyond the patient population in which the company reported data for its randomized clinical trials. It includes a full approval for Duchenne patients who are not dependent on a wheelchair, and an accelerated approval for those who are wheelchair-dependent. Marketed as Elevidys, the gene therapy was initially given accelerated approval last year for boys ages 4 and 5 who have Duchenne muscular dystrophy. It’s meant to slow progression of the aggressive muscle disease, and the expansion to older boys has long been awaited by families of patients who have few treatment options. It costs $3.2 million in the US and is not approved anywhere else in the world. There have been signs the FDA was going to expand the therapy’s label. Earlier this year, CBER Director Peter Marks made comments suggesting he was open to doing so. And when Elevidys was initially approved, Marks overruled FDA reviewers who had recommended rejecting it. Marks again overruled FDA staff in broadening the label of Sarepta’s gene therapy. Memos that were released Thursday with the agency’s decision showed that two top FDA officials, Lola Fashoyin-Aje and Nicole Verdun, recommended rejecting the gene therapy in favor of an additional confirmatory study but were overruled by CBER chief Peter Marks. It’s been debated whether the evidence has been strong enough to back the expansion. In October, the company reported that the Phase 3 EMBARK study failed to meet its primary endpoint. Intended as a confirmatory study, it showed that boys ages 4 to 7 who got Elevidys didn’t do better on a 17-item motor skill test than those who got placebo. However, the study met key secondary endpoints — measuring boys’ ability to stand and walk — that Sarepta cited when asking the FDA to approve the therapy for boys with Duchenne regardless of their wheelchair status or age. Continued approval for non-ambulatory Duchenne patients may depend on results from a confirmatory trial, which Sarepta said would be the Phase 3 ENVISION study with non-ambulatory and older ambulatory people with Duchenne. In its first six months on the market, Elevidys generated $200 million in revenue, reflecting strong demand for the therapy. The company’s stock $SRPT was up 35% in after-hours trading. It’s also been helped by the failure of a potential competitor: Last week, Pfizer announced that its Duchenne gene therapy did not succeed in a Phase 3 study. Pfizer’s therapy did not meet any of the secondary endpoints that Sarepta pointed to when asking for its label expansion. #lucidquest #genetherapy #celltherapy